Follow
'Even within one rare disease, many subtypes can exist that require personalized approaches to therapy. As one example, EMA’s chief medical officer Steffen Thirstrup mentions cystic fibrosis, which affects around 1 in 2,500 babies born. “When I left medical school 30 years ago, we knew that cystic fibrosis was caused by a genetic defect, but now we know that different mutations in the genome can cause this disease,” he explains.'
#raredisease
